A six-month double-blind controlled study was performed to determine effectiveness and dose response of disodium etidronate on Paget's disease (osteitis deformans). Forty-seven subjects were studied with pain related to radiologically documented Paget's bone sites as well as marked elevation of serum alkaline phosphatase and hydroxyproline excretion. There was significant (p<0.05) reduction of alkaline phosphatase, hydroxyproline and radiofluoride or radiostrontium uptake over Paget's bone lesions after orally administered disodium etidronate at doses of 5, 10 and 20 mg per kilogram per day for six months. There was a dose-response relation of administered disodium etidronate to improvement of pain, with moderate or marked improvement of pain in six of seven subjects on 20 mg per kilogram per day. In all dosage groups the most marked relief of pain was at sites of Paget's involvement of long bones. Apparent side effects were limited to occasional abdominal cramps. (N Engl J Med 289:1379–1384, 1973).
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